Novel capsid technology to enable convenient delivery in the doctor’s office of IKAR-003 for intermediate AMD, designed to prevent progression to sight-threatening disease

NORWICH, UK & CHICAGO, US, January 6 2026 – Ikarovec Ltd, which is developing dual-pathway gene therapies for vision-threatening retinal diseases, and VectorBuilder, a global leader in gene delivery technologies, have entered into an exclusive worldwide option agreement for VectorBuilder’s novel AAV capsid technology to be used in combination with Ikarovec’s gene therapy candidate IKAR-003 for intermediate age-related macular degeneration (AMD). Following successful further evaluation of the technology, the companies will engage in a strategic partnership under which Ikarovec will be responsible for clinical development and commercialisation of IKAR-003. Based on the potential of IKAR-003 in intermediate AMD, the proposed deal is expected to be worth in excess of $1bn.

VectorBuilder’s proprietary capsid technology will allow IKAR-003 to be delivered to the eye via the minimally invasive intravitreal route in a doctor’s office. This will enhance access to the treatment for the target patient population – those at the earlier, intermediate AMD stage for whom prevention of disease progression is a priority. IKAR-003 is designed to prevent this progression through a one-time injection of an AAV-delivered dual-pathway gene therapy combining neuroprotection and complement modulation to preserve visual function.

Patients with intermediate AMD, a condition which affects millions worldwide and currently has no approved drug treatments, are at significant risk of progressing to geographic atrophy or wet AMD, both of which cause irreversible vision loss.

‘Partnering with VectorBuilder on IKAR-003 increases the value of Ikarovec’s pipeline by creating an office-based intravitreally delivered product, ideal for large-scale adoption in the prevention of disease progression setting.’ said Thomas Ciulla, MD, MBA, President and CEO of Ikarovec. ‘We have assessed other intravitreal capsid options and are confident that VectorBuilder’s technology will provide superior efficacy.’

‘IKAR-003 uses the same dual-pathway approach as our lead programme, IKAR-001, which remains on track for clinical trial initiation in late 2026 in geographic atrophy, using validated subretinal delivery in this advanced stage of disease. We are therefore addressing two distinct patient populations with a delivery approach tailored to each.’ he added.

‘We have worked with Ikarovec for several years and have developed a deep understanding of the company’s approach and technical requirements’. said Dr. Bruce Lahn, Founder and Chief Scientist at VectorBuilder. ‘By utilizing our AI-powered DeepCap platform that integrates rational design with ultradeep search of AAV sequence space, we were able to engineer best-in-class ocular capsids. In non-human primate studies, our intravitreally administered capsids can target wide areas of the retina and successfully transduce virtually all cells of the macula, demonstrating broader and more robust transduction than current clinical intravitreal capsids. We are confident that our capsid technology, combined with Ikarovec’s innovative dual-pathway therapeutic approach and experienced leadership team, will create a strong foundation for developing transformative therapies for patients with vision-threatening retinal diseases.’

–ENDS—

For further information, please contact                      

Emma Palmer Foster, Communications                                + 44 (0)7880 787185

comms@ikarovec.com

Notes for Editors

About Ikarovec

Ikarovec Limited is a UK-based, near clinical stage biotechnology company pioneering dual-pathway gene therapies for vision-threatening retinal diseases. Founded in 2020 as a spin-out from Quethera (acquired by Astellas Pharmaceuticals in 2018), the company is headquartered at Norwich Research Park in Norfolk, United Kingdom. Ikarovec’s proprietary bicistronic gene therapy platform builds on validated therapeutic approaches and adds synergistic second mechanisms, addressing what current single-pathway therapies miss with reduced development risk. The company’s lead programmes address large market opportunities including geographic atrophy and retinitis pigmentosa (using validated subretinal delivery) plus intermediate AMD and wet AMD (via doctor’s office-based intravitreal delivery). Ikarovec is led by an experienced team with expertise in bicistronic gene therapies and direct involvement in major ophthalmology products and exits totaling over $13 billion including Spark, Iveric and EyeBio. For more information, visit www.ikarovec.com and follow us on LinkedIn.

About VectorBuilder

VectorBuilder is a global leader in gene delivery technologies. As a trusted partner for thousands of labs and biotech/pharma companies across the globe, VectorBuilder offers a full spectrum of gene delivery solutions covering virtually all research and clinical needs from bench to bedside. The company’s R&D programs have focused on enhancing the functionality, safety and manufacturability of genetic medicines by solving major pain points in current gene delivery technologies such as low tissue specificity, immunogenicity, limited cargo size, and low manufacturing yield. VectorBuilder’s proprietary DeepCap platform combines machine learning and rational design with massively parallel ultradeep search of distributed sequence space to power the engineering of novel AAV capsids with best-in-class transduction efficiency and tissue specificity. Utilizing this platform, VectorBuilder has built a large panel of capsids targeting a wide range of therapeutically important tissues including CNS, PNS, muscle, heart, eye and ear. The company also operates about 10,000 m² of modern GMP facilities for genetic medicine manufacturing, fully compliant with ICH guidelines and US and EU regulatory standards. VectorBuilder has provided IND-enabling vectors to a global client base across the US, Europe, Australia, Japan, China, and South Korea. The company has been honored with numerous awards such as BioTech Breakthrough Award, PharmaVoice 100 Award, Bioz Rapid Star Award, and CDMO Leadership Award. For more information, visit www.vectorbuilder.com.

International experts to help drive company success as it launches its Series A fundraising

Norwich, UK, September 30 2025 – Ikarovec, a leader in ophthalmology gene therapy innovation with a sophisticated field-changing technology for the long-term relief of prevalent eye diseases, has established a world-leading Scientific Advisory Board (SAB) of retinal research and clinical care specialists. The SAB, comprising a Clinical Translation Council and a Discovery Research Council, will advise the company across its activities from preclinical research through to clinical trials, regulatory affairs and key opinion leader engagement. Ikarovec has recently launched its >$40m Series A round to take its lead product, IKAR-001 for geographic atrophy (GA) with a secondary indication of retinitis pigmentosa (RP), through Phase I and II trials and to advance the pipeline.

Bringing together clinical retina specialists, vision scientists and gene therapy experts, the SAB has a strong international perspective including members from Australia, Israel, the UK and the US. With over 180,000 citations and in excess of 2,500 publications, it has exceptional scientific credibility plus clinical trial and regulatory approval experience.

IKAR-001, the first product from Ikarovec’s bicistronic gene therapy platform, takes a differentiated dual-pathway approach combining neuroprotection and complement modulation in a single treatment – unlike current approved GA therapies that target complement alone and require regular injections. This approach is designed to preserve vision rather than just slow its loss, while IKAR-002 for wet age-related macular degeneration combines anti-angiogenic and anti-fibrotic mechanisms. Experts on the SAB will bring further academic rigour and clinical development expertise in these areas.

Dr Thomas Ciulla, MD, MBA, President & CEO of Ikarovec, said, “To have such a stellar group of world leaders in retinal research and clinical care choosing to join our Scientific Advisory Board is both a validation of the potential of our bicistronic gene therapy approach and a great honour. We are delighted to have access to this level of expertise as we advance our bicistronic gene therapies with precision, innovation, and a commitment to addressing ophthalmic diseases that cause significant sight loss.”

The SAB¹ Clinical Translation Council members are:

• Robert L. Avery, MD – Founder and CEO of California Retina Consultants
• Dean Eliott, MD – Director Emeritus of the Mass Eye and Ear Retina Service and Professor of Ophthalmology at Harvard Medical School  
• M. Dominik Fischer, MD, DPhil – Professor of Ophthalmology at University of Oxford, Centre for Ophthalmology Tübingen & University of Lausanne, Consultant Ophthalmic Surgeon at Oxford Eye Hospital
• Robyn H. Guymer, MD, PhD – Professor of Ophthalmology at the University of Melbourne and Deputy Director of the Centre for Eye Research Australia (CERA)
• Allen C. Ho, MD, FACS – Attending Surgeon and Director of Retina Research at Wills Eye Hospital and Professor of Ophthalmology at Sidney Kimmel Medical College at Thomas Jefferson University, Past President of the Retina Society
• Anat Loewenstein, MD, MHA – Professor & Director of the Division of Ophthalmology at Tel Aviv Medical Center and Sydney Fox Chair of Ophthalmology at Tel Aviv University’s Sackler Faculty of Medicine, President EURetina
• Robert E. MacLaren, MD, DPhil -Professor of Ophthalmology at University of Oxford and Consultant Ophthalmologist at Oxford Eye Hospital, UK NIHR Senior Investigator
• Carl D. Regillo, MD, FACS – Director Retina Service Wills Eye Hospital & Professor of Ophthalmology, Sidney Kimmel Medical College, Thomas Jefferson University
• Charles C. Wykoff, MD, PhD – Director of Research at Retina Consultants of Texas & Chairman of Research for Retina Consultants of America, Deputy Chair of Ophthalmology at Houston Methodist Hospital, Past President Vit-Buckle Society

The SAB¹ Discovery Research Council members are:

• Christine A. Curcio, PhD – Professor Emerita of Ophthalmology and Visual Sciences at the University of Alabama at Birmingham
• Rajendra Kumar-Singh, PhD -Professor of Developmental, Molecular and Chemical Biology at Tufts University School of Medicine

–ENDS—

¹ For full biographies and photos, please go to https://ikarovec.com/team/

For further information, please contact                      

Emma Palmer Foster, Communications                                + 44 (0)7880 787185

comms@ikarovec.com

Notes for Editors

About Ikarovec

Ikarovec is a leader in ophthalmology innovation, with a sophisticated field-changing technology for the long-term relief of prevalent eye diseases. The company’s flexible proprietary bicistronic gene therapy generates multiple synergistic therapeutic proteins where they are needed in the eye, bringing a differentiated approach to treating a range of large and growing ophthalmic disorders.

Its lead candidate, IKAR-001 (previously known as IKC159V), will proceed to geographic atrophy (GA) and gene-agnostic retinitis pigmentosa (RP, secondary indication) clinical trials supported by studies showing preservation of cells critical for vision. GA and RP lead to significant vision loss, affecting 6.5 million people worldwide. There are very limited treatment options for RP whilst current treatment options for GA (only approved in the US, Japan & Australia) require regular injections directly into the eye. Ikarovec’s novel bicistronic gene therapy leads to the expression of two therapeutically relevant proteins, targeting multiple causes of the disease with a single injection which is expected to be disease modifying. Follow-on programmes are targeting wet age-related macular degeneration (AMD), intermediate age-related macular degeneration (iAMD) and other chronic poorly managed ophthalmic indications.

Ikarovec is directed by an international Board and management team with industry-leading ophthalmology and gene therapy expertise. This experience comes via Astellas, EyeBio, OXB (Oxford Biomedica), Spark Therapeutics and others. Ikarovec, which is based at the Norwich Research Park, is backed by LifeArc Ventures, Parkwalk Advisors and UKI2S. More information at www.ikarovec.com and on LinkedIn.

Posted on 28th May 2025 by ikarovec

• Studies presented at leading ophthalmology and gene therapy conferences demonstrate therapeutic potential in geographic atrophy and wet AMD, leading causes of legal blindness
• This single-administration gene therapy approach could replace frequent eye injections for geographic atrophy and wet AMD patients
• The lead candidate IKAR-001 (previously known as IKC159V) will proceed to a geographic atrophy clinical trial supported by studies showing preservation of cells critical for vision

Norwich, UK, May 28 2025 – Ikarovec, a leader in ophthalmology innovation with a sophisticated field-changing technology for the long-term relief of prevalent eye diseases, has highlighted the therapeutic potential of its approach in geographic atrophy and wet AMD, with presentations at major ophthalmic and gene therapy conferences. The new preclinical data discussed at ARVO¹ and ASGCT² show how the company’s flexible bicistronic gene therapies can generate multiple therapeutic proteins where they are needed in the eye, protecting retinal cells and preventing damaging blood vessel growth and scarring. 

The lead candidate IKAR-001 will proceed to a geographic atrophy clinical trial supported by studies showing preservation of cells critical for vision. The preclinical studies presented on the investigational agent, which encodes the therapeutically relevant proteins pigment epithelium-derived factor (PEDF) and soluble CD46, demonstrated its potential to improve clinical outcomes in geographic atrophy, an advanced common form of AMD.  

Additional studies presented at the conferences showed how another of the company’s bicistronic gene therapies reduced damaging new blood vessel formation, vascular leakage and scarring in preclinical models of wet AMD. Encoding inhibitors of vascular endothelial growth factor (VEGF) and connective tissue growth factor (CTGF), two proteins involved in driving disease progression and scarring respectively, this dual-target approach could enhance outcomes in wet AMD sufferers. 

Dr Thomas Ciulla, President & CEO of Ikarovec, said, “Sufferers of devastating eye diseases such as geographic atrophy and wet AMD are in real need of effective disease modifying treatments. The new preclinical data we have presented shows the potential of Ikarovec’s proprietary bicistronic gene therapy approach to generate these improved outcomes for patients, producing multiple therapeutic proteins in the eye to protect cells and reduce damage.”

“In addition, our approach aims to dramatically reduce treatment burden on patients, by replacing frequent, often monthly, eye injections with a single administration, potentially improving both compliance and long-term outcomes,” he added. “Encouraged by the evidence generated to date and supported by our recent fundraising, we are working to complete advanced preclinical studies with our lead candidate IKAR-001, ready for the milestone of a clinical trial.”

–ENDS—

¹ The Association for Research in Vision and Ophthalmology; abstracts 3763 & 4271

² American Society of Gene & Cell Therapy; poster numbers 573 & 574

For further information, please contact                            

Emma Palmer Foster, Communications                          + 44 (0)7880 787185

comms@ikarovec.com

Posted on 6th May 2025 by ikarovec

Spark Therapeutics veteran brings over 25 years’ of leadership experience to drive translation of company’s bicistronic gene therapies into products

• Ikarovec, which is developing field-changing bicistronic gene therapies for ophthalmic diseases, appoints Dr Thomas Ciulla, expert in ophthalmology drug development, as President and CEO.
• Dr Ciulla directed medical strategy for Luxturna, the first FDA-approved gene therapy for inherited retinal disease, at Spark Therapeutics, with other development achievements at Viridian Therapeutics, Iveric Bio and Clearside Biomedical. 
• Dr Ciulla will drive Ikarovec’s pipeline forward as it transitions into a clinical phase company, with its first trial expected to be of lead product IKC159V in geographic atrophy (GA), a common and severe form of age-related macular degeneration.

Norwich, UK, May 6 2025 – Ikarovec, a leader in ophthalmology innovation with a sophisticated field-changing technology for the long-term relief of prevalent eye diseases, is pleased to announce that Thomas Ciulla MD MBA, a leader in ophthalmology drug development, has been appointed President and CEO.  He joins the company from Viridian Therapeutics, where he was Chief Medical Officer and accelerated veligrotug (for thyroid eye disease) from Phase II to two successful Phase III pivotal trials.

Dr Ciulla’s other significant activities include direction of medical strategy for Luxturna, the first FDA-approved gene therapy for inherited retinal disease, at Spark Therapeutics prior to its acquisition by Roche. Also notable is his involvement in the first successful Phase III trial in age-related macular degeneration/GA, leading to US approval of Izervay at Iveric Bio (now part of Astellas). As Chief Medical Officer at Clearside Biomedical, he contributed to development of the first FDA-approved suprachoroidal therapy (Xipere) and to key industry partnerships, including a collaboration for suprachoroidal delivery of gene therapy, demonstrating a track record in facilitating strategic alliances and financing. 

Dr Ciulla is a Harvard-trained board-certified ophthalmologist and retina specialist. As a clinical researcher with over 300 publications, Dr Ciulla brings substantial academic credibility and research expertise in retinal diseases to Ikarovec’s innovative platform.

Ikarovec’s lead product, IKC159V, a bicistronic gene therapy for GA, is expected to be the company’s first candidate to reach the clinic. GA is a leading cause of significant vision loss and affects 5 million people worldwide. The company’s flexible proprietary bicistronic gene therapy platform generates multiple synergistic therapeutic proteins where they are needed in the eye, bringing a differentiated approach to treating a range of large and growing ophthalmic disorders. 

Dr Thomas Ciulla, President and CEO of Ikarovec, said, ‘I’m pleased to be joining Ikarovec at such an exciting phase of its development, as it transitions to a clinical phase company. With more than 5 million people worldwide affected by geographic atrophy alone, I’m committed to advancing our bicistronic gene therapy platform which targets multiple disease pathways through a single administration, potentially improving outcomes while significantly reducing the treatment burden compared to current therapies. I’m looking forward to helping translate this innovative technology into products which provide meaningful clinical benefits for those facing vision loss.’

Dr David Guyer, Chairman of Ikarovec, added, ‘With a track record spanning innovation in ophthalmology and gene therapy, and a proven background of FDA approvals and value creation in companies, Tom is a great asset to the team and I’m delighted to welcome him to Ikarovec. He will be pivotal to driving our pipeline forward, leading partnering and fundraising, and establishing our US presence.’

–ENDS—

Photo available on request

For further information, please contact                            

Emma Palmer Foster, Communications                          + 44 (0)7880 787185

comms@ikarovec.com

Introduction

Funds raised to continue advanced pre-clinical trials of novel gene therapies to treat blindness and major chronic eye disease

Led by LifeArc Ventures and Parkwalk Advisors, with participation from the UK Innovation, Science and Seed Fund, managed by Future Planet Capital (UKI2S)

Dr Jean-Philippe Combal of Vivet Therapeutics and SpliceBio appointed as NED

Norwich, UK, October 23 2024 – Ikarovec, which is developing novel bicistronic gene therapies for major chronic eye diseases, is pleased to announce that it has extended its seed funding round by £5M, bringing the total raised to £8M. The financing was led by existing investors LifeArc Ventures and Parkwalk Advisors, with UKI2S (managed by Future Planet Capital) also participating in the round. The company is moving towards IND-enabling studies for its lead programme, IKC159V, in geographic atrophy (GA), which causes major sight loss in millions of people globally.The funds raised will enable the company to continue advanced preclinical studies with IKC159V, a novel bicistronic gene therapy that secretes two clinically relevant proteins that reduce overall oxidative stress and inflammation, key hallmarks of GA, in one injection. They also protect the retinal cells from atrophy and reduce the overall risk of conversion of GA to wet age-related macular degeneration. Other preclinical work and manufacturing process development will also take place with the funding.IKC159V has shown robust and differentiated data in multiple disease relevant models, with superior efficacy to single gene therapies in comparative studies. Ikarovec has demonstrated that the gene therapy protects key retinal cells from damage whilst minimising vessel leakage and instability, processes that lead to disease progression.Dr Katie Binley, CSO of Ikarovec, said ‘We’re excited by the progress we’ve made with our lead programme for geographic atrophy and are advancing it towards the clinic as quickly as possible. Ikarovec would like to thank its investors for their continued support to enable us to do this and to advance our pipeline of gene therapies for major eye diseases.’Dr David Guyer, Chairman of Ikarovec, added ‘Ikarovec’s approach of expressing clinically relevant and synergistically acting proteins via bicistronic vectors differentiates it in the ophthalmic gene therapy area. The company is grateful for the ongoing recognition of its potential by LifeArc Ventures, Parkwalk Advisors and UKI2S as we develop important new products for devastating eye conditions.

In addition, the company is excited to welcome Dr Jean-Philippe Combal of Vivet Therapeutics and SpliceBio to the Board as a Non-Executive Director. He brings extensive experience in both ophthalmic product development and gene therapy through positions at GenSight Biologics and Sanofi-Fovea.’

For further information, please contact
Emma Palmer Foster, + 44 (0)7880 787185
Communications comms@ikarovec.comn